The FDA is Not the Enemy

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“…FDA has demonstrated its commitment to supporting access to experimental therapies for patients living with serious diseases. The agency is an important part of the process, helping to ensure that patients are protected from potentially harmful drugs or one that doesn’t work. Allowing access to investigational therapies requires the cooperation of others, including drug companies and doctors, not just FDA. In cases where a company has initially denied access, FDA has stepped in to help. FDA is proud of its record facilitating access to safe, effective therapies for patients who need them as quickly as possible.”

-Margaret Hamburg: Commissioner, U.S. FDA

This is from the first written comment that Dr. Hamburg has made with respect to the Right to Try (RTT) bill, recently signed into law in Colorado, now making its way across the country. To those of you who follow this area, it may surprise you to know that Neuralstem agrees with Commissioner Hamburg; the FDA is an indispensable party in this process. Indeed the RTT bill relies heavily on their expertise and diligence. That is why the bill only applies to fatal diseases, and only to drugs or therapies that have passed an FDA-approved safety trial, and are still actively being developed under the FDA umbrella in further trials. Any drug that dropped out of this legislative “inclusion” criteria would no longer be eligible for delivery under the legislation. At Neuralstem we are involved in developing our cell therapies all over the world, and I can attest to the fact that the FDA’s science and processes are considered the gold standard. Again, this is exactly why this important safeguard is incorporated into the RTT bill.

Commissioner Hamburg accurately notes that giving access to therapies requires all players in the medical field to work together, including pharmaceutical companies. Often that lack of access can be attributed to a Company’s various fears and issues with providing that access. And again, we believe that RTT can be a bridge over that chasm. As it is enacted into law, and carries the weight of law, everyone involved will be more comfortable with the process; and in turn start to address the issues involved in implementing early access programs. These types of programs are carried out all over the world in various formats. None of those has the safeguards that RTT is able to rely on by being restricted to FDA-approved testing.

As Commissioner Hamburg states, the problem of increasing early access to experimental drugs for dying patients requires an industry wide response. At Neuralstem, we see 5,000 ALS patients diagnosed each year, yet our Phase I and Phase II trials combined will only have included 30 patients. On average these patients die two to four years from diagnosis. Thousands have contacted us about possible inclusion in the trials. RTT is a scaffold on which an industry wide national infrastructure can be built, state by state if that’s what it takes, to help patients across the spectrum of fatal diseases participate actively in trying to find a cure for their disease; and become active partners in the process rather than passive victims.

No one is suggesting that this process take the place of the existing clinical trial process. And no company (though I can only speak for Neuralstem) will offer a drug if the FDA tells it not to. The heart of the RTT bill recognizes the contribution the FDA process makes to the determination of safety in the early stages of drug development and Neuralstem’s support of this program is based upon that recognition.

No one is promising cures. Every patient that Neuralstem can treat under this law until we have FDA approval will go through the exact same informed consent process that patients in our trials go through. They will understand the risks. No matter how encouraging the early public information may be, all of our patients understand that it is too soon and there have been too few patients for us to know that it can help them. Dying patients deserve the unvarnished truth, and, in our experience, they demand it.

Supporters of RTT are not suggesting that the FDA is not doing all it can. However, their existing programs do not have to be the only tools available to patients fighting for their lives. RTT is not “the answer,” But it is a very important and fundamental piece of a new structure that we believe can have a serious impact on expanding early access to experimental drugs for dying patients. We look forward to working with the FDA and anyone else who is interested in helping to expand access to experimental drugs to patients who have been diagnosed with fatal diseases.

Commissioner Hamburg has stated in no uncertain terms that the FDA is committed to this concept. I for one, believe her. The FDA is not the enemy- fatal diseases are the enemy, and we are all on the same side in the fight against them.

PS: many of you have written and asked about the possible timing of the effort in Colorado. We simply do not know, but it is unlikely to be in the immediate future. Our therapy is not a “pill” which can simply be sent to doctors to administer. It is an invasive surgery and we will have to identify and train a team, just like we did when recruiting Emory, Michigan and MGH to be sites for the trial. This involves observing human surgeries in the existing trial, and then practicing with the surgical device. It will also involve identifying a center that will participate, and educating the ALS clinicians in the State about our therapy. So as you can see, it is an involved process; but we are committed to pushing it forward and as always we are working as hard and as fast as we can.

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This blog may contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this blog regarding potential applications of Neuralstem's technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem's periodic reports, including the annual report on Form 10-K for the year ended December 31, 2012 and the Form 10-Q for the period ended June 30, 2013.

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